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Issues in the design of new clinical trials for rheumatoid arthritis therapeutics.

机译:类风湿关节炎治疗新临床试验设计中的问题。

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摘要

Rheumatoid arthritis (RA) is a chronic, heterogeneous disease, for which there has traditionally been few reliable prognostic indicators or simple clinical outcome measures with which to adequately assess disease status or clinical improvement. These challenges have hindered the assessment of new therapeutic agents. Investigators in rheumatology have, therefore, attempted over the past two decades to develop new approaches and scoring systems to facilitate the study and development of therapies. As a result of these efforts, the efficacy of almost all new agents tested in clinical trials are now assessed with widely accepted scoring systems that measure improvement in three important areas. First, changes in patient signs and symptoms are measured via composite indices of both clinical and laboratory parameters. Second, the progression of structural damage is assessed by radiographic imaging. Finally, long-term improvement in patient function is measured via patient-reported outcomes. These changes to clinical trial design have been adopted by health authorities around the world. Further challenges, however, must be overcome in order for progress to continue.
机译:类风湿关节炎(RA)是一种慢性异质性疾病,传统上,对于这种疾病,可靠的预后指标或简单的临床结果指标很少能充分评估疾病状况或临床改善。这些挑战阻碍了对新治疗剂的评估。因此,风湿病学研究人员在过去的二十年中尝试开发新的方法和评分系统,以促进疗法的研究和开发。这些努力的结果是,现在已使用广泛接受的评分系统评估了在临床试验中测试的几乎所有新药物的功效,这些评分系统可衡量三个重要领域的改善情况。首先,通过临床和实验室参数的综合指数来测量患者体征和症状的变化。其次,通过射线照相成像评估结构损伤的进展。最后,通过患者报告的结果来衡量患者功能的长期改善。对临床试验设计的这些更改已被世界各地的卫生当局采用。但是,必须克服进一步的挑战,才能继续取得进展。

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